Kicking Off GM1 Day 2023!

A worldwide movement, GM1 Awareness Day was started to provide a rallying point for our community to spread awareness about and to advocate for all those living with GM1. We want as many people as possible to know about this devastating and fatal disease so we can increase funding for research and drug development. Our hope is that progress in those areas will advance the creation of effective treatments & therapies and ultimately – save lives. 

If you want the same thing, help us! From now until GM1 Day, May 23rd, there will be lots of opportunities to participate and spread awareness. Now is the time to order GM1 Day gear, plan fundraisers, and reach out to local media. Plan on wearing blue, navy, and yellow and holding up the number one online! 

Cure GM1 Met With the FDA

Cure GM1’s President, Christine Waggoner, along with 11 other rare disease patient advocates met with the FDA commissioner and the directors of the FDA. During this meeting, she shared the status of GM1 drug development and reiterated the desperate need for an approved therapy for GM1. With the halting of several clinical trials and closures of biotech companies, the FDA is in a critical position to recognize how it could be more accommodating to rare diseases. We must continue to push for meaningful change for the GM1 community and for all rare diseases. 

We feel privileged to have been able to participate. Thank you Emil Kakkis for organizing the meeting, to all the amazing advocates, and to the FDA staff for joining to discuss the overall rare disease landscape.

News

Violet’s family advocates for statewide GM1 Gangliosidosis Awareness Day in California.  Violet’s family was also featured in the a UC Berkeley Alumni feature.

Lysogene announces that no takeover offer was filed. This sad news means the termination of all their programs, including their GM1 gene therapy program.

The Azafaros PRONTO Natural History Study webinar is now up on our YouTube. Please watch to learn more about the importance of natural history and how families can help support this study. Make sure to subscribe so you know when we upload new videos!

Community Efforts

Thank you to A Cure for Marley for hosting the Face Off For Marley Charity Hockey Game to spread awareness for GM1. What a special experience for everyone involved! Our mission could not be what it is without families like yours. 

Dean’s family was featured in the article and video: All this can be avoided with a genetic test; it’s hard to see your son regressing on TVM news. Thank you so much for reaching out to local media to share your story. Your advocacy makes a huge difference. 

Luigi’s story is now on the Cure GM1 Website. Learn about this sweet boy and his incredible strength. Also consider sharing your story to help us spread awareness about GM1 gangliosidosis so we can make progress toward effective treatments for children like Luigi.

May is Mental Health Awareness Month

We encourage all GM1 Caregivers to take time to care for themselves. You deserve all the care you give to your loved ones — remember, you won’t be able to support anyone without first supporting yourself. Good self care routines include getting sunlight outside first thing in the morning, scheduling alone time for yourself, meditating, journaling, and taking walks. We believe in you. 

Support Cure GM1

Spring Support

  • Sow seeds of hope when you sow seeds in your garden! Help advance research into possible treatments for GM1 when you purchase veggies, fruits, and beautiful bulbs and seeds. Fifty percent of net proceeds will be donated. Order before May 15th!
  • Now is your last chance to order Mother’s Day Cards as well as Mother’s Day t-shirts. Order yours now!

Ways to Give – Your Support Matters

Charity Miles | RoundUp App | Walmart RoundUP | Facebook Fundraisers | Donate | Set up a Recurring Donation

Visit our Take Action page for more ways to support our community